Duchenne muscular dystrophy
RSS feedNo additional long-term beneficial effects for the bisoprolol-perindopril combination
British clinicians report the long-term results of a prophylactic treatment for cardiomyopathy in Duchenne muscular dystrophy (DMD): the combination of perindopril (a conversion enzyme inhibitor) and bisoprolol (a beta-blocker) had already been the focus of an initial randomised clinical trial by the same team in 2011 in children with DMD aged between 5 and 13 … [Read more]
Towards conditional authorisation of givinostat in Europe for walking DMD patients aged 6 and over on corticosteroids
Givinostat, a histone deacetylase (HDAC) inhibitor that acts on muscle fibrosis and inflammation in Duchenne muscular dystrophy, should be authorised in Europe very soon, under the name Duvysat™ (it has been authorised in the United States since March 2024). The application submitted to the EMA by the Italfarmaco laboratory was examined by the Committee for … [Read more]
Positive results with gene therapy in a monkey model of DMD
A Chinese team has developed a rhesus monkey model of Duchenne muscular dystrophy, enabling it to evaluate a new gene therapy, with encouraging results. The DMDEx50 animal model has mutations in exon 50 of the DMD gene. A single-vector gene therapy called MyoAAV/Cas12iMax/sgRNA3Ex51 targeting exon 51 of the DMD gene was developed using Cas12iMax technology … [Read more]
Overexpressing utrophin in DMD: a new therapeutic approach in the spotlight
Chinese researchers have revived a therapeutic technique designed to over-express utrophin, an endogenous protein very similar to dystrophin: they used a genome-editing approach in several models (cellular and animal) of Duchenne muscular dystrophy (DMD) combined with a Myo-AAV muscle-specific viral vector (to obtain MyoAAV-UA). after administration, robust and long-lasting overexpression of utrophin was obtained in … [Read more]
A significant advance in DMD cell therapy
British researchers have succeeded in significantly improving the grafting of myogenic progenitor cells (MPC) into the muscle fibre of mouse models of Duchenne muscular dystrophy: they used a special gel (hydrogel) to stabilise the donor cells so that they would take better hold in the muscle of the recipient mice, Stem cells from patients suffering … [Read more]
Valproic acid improves exon skipping efficiency in DMD mouse models
The major challenge of exon skipping therapy in Duchenne muscular dystrophy is to effectively deliver the antisense oligonucleotides to the targeted tissues, in this case the muscles. After obtaining encouraging results over four weeks, a French team assessed the benefits of combining valproic acid with antisense oligonucleotides designed to skip exon 23 of the DMD … [Read more]
Tracking and preventing possible myocardial complications: a major challenge for DMD gene therapy
As treatment with delandistrogene moxeparvovec begins to be widely prescribed in the United States for Duchenne muscular dystrophy (DMD), American experts have come together to issue recommendations aimed at better identifying and preventing the deleterious effects of this gene therapy (GT) on the heart: cases of myocarditis induced by TG remain exceptional but can be … [Read more]
International recommendations for the child-adult transition in DMD
A group of international experts has drawn up international recommendations concerning the transition of patients with Duchenne muscular dystrophy (DMD) from paediatric to adult age: in real life, this pivotal period is often synonymous with loss of follow-up, which can be detrimental to the functional future of the DMD patient, Even though feedback varies from … [Read more]
Positive phase I/II results for brogidirsen in Duchenne muscular dystrophy
Brogidirsen is a dual-targeting phosphorodiamidate morpholino oligomer (PMO) antisense oligonucleotide composed of two sequences targeting exon 44 of the DMD gene in Duchenne muscular dystrophy. A Japanese phase I/II clinical trial involving six patients aged between 4 and 13 years demonstrated its ability to partially restore dystrophin expression. Of the six patients, five had a … [Read more]
DMD: an effective gene therapy in a severe mouse model of the disease leads to cardiac inflammation
Duchenne muscular dystrophy, a severe and progressive hereditary muscular dystrophy, is caused by mutations in the DMD gene leading to the loss of a protein essential for muscle function, dystrophin. There is currently no effective treatment, but gene therapy trials using micro-dystrophins have been underway for several years. These strategies, targeting both skeletal and cardiac muscle, are applicable to all … [Read more]